EU Approval Saga of PTC's DMD Therapy Translarna Continues

PTC Therapeutics announced that it intends to request another re-evaluation of its conditional marketing authorization for Translarna (ataluren), a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD), after the European Medicines Agency (EMA) maintained its unfavorable stance. This announcement resulted in a 13% drop in the company’s shares.

The Committee for Medicinal Products for Human Use (CHMP) of the EMA had re-examined the data supporting Translarna at the behest of the European Commission. The CHMP was instructed to review the entirety of the evidence, which included data from patient registries and real-world evidence.

Despite this review, the EMA concluded that the additional evidence presented was insufficient to warrant a positive opinion on Translarna. The CHMP pointed out that the meta-analysis conducted on three clinical studies had several flaws and that the results were not compelling enough to override the negative outcomes of the individual trials. Furthermore, the EMA stated that no definitive conclusion on the efficacy of Translarna could be drawn from the real-world data available.

Translarna originally received conditional approval in Europe a decade ago to treat nmDMD in ambulatory patients aged five and older. This approval was later expanded to include children as young as two years old. However, last year, when the annual renewal of Translarna’s marketing authorization was under review, the CHMP rendered a negative opinion, which PTC subsequently appealed.

In January, the CHMP reaffirmed its negative recommendation against Translarna. Nonetheless, the European Commission did not adopt this decision. On Friday, PTC stated that the marketing authorization for Translarna is still in effect, and the therapy is expected to remain available on the market until the end of 2024, even if the negative opinion is upheld and ratified by the European Commission.

RBC Capital Markets analyst Brian Abrahams commented on the development, noting that the CHMP’s opinion will now be forwarded to the European Commission for a final, legally binding decision. Abrahams remarked that the regulator’s negative stance perpetuates uncertainty around Translarna in Europe and could potentially add risk to its future in the US.

In March, PTC detailed its plans to re-submit a marketing application for Translarna to the FDA by mid-year, following feedback from the agency. The FDA had previously rejected an application for Translarna in 2017, citing the need for an additional adequate and well-controlled trial to confirm the treatment’s benefits.

This series of events highlights the ongoing challenges PTC Therapeutics faces in securing unwavering regulatory approval for Translarna, a critical therapy for patients with nmDMD. As the company navigates the complex landscape of regulatory requirements both in Europe and the US, the future of Translarna remains in a state of flux. However, PTC's commitment to pursuing further evaluations and submissions underscores its dedication to providing this treatment to patients who need it.

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